EP3863645A4 - Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders - Google Patents
Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders Download PDFInfo
- Publication number
- EP3863645A4 EP3863645A4 EP19861226.9A EP19861226A EP3863645A4 EP 3863645 A4 EP3863645 A4 EP 3863645A4 EP 19861226 A EP19861226 A EP 19861226A EP 3863645 A4 EP3863645 A4 EP 3863645A4
- Authority
- EP
- European Patent Office
- Prior art keywords
- treatment
- modified mrna
- intrahepatic cholestasis
- progressive familial
- familial intrahepatic
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Genetics & Genomics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Toxicology (AREA)
- Zoology (AREA)
- General Chemical & Material Sciences (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physics & Mathematics (AREA)
- Biotechnology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Dermatology (AREA)
- Inorganic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862731055P | 2018-09-13 | 2018-09-13 | |
| US201962840369P | 2019-04-29 | 2019-04-29 | |
| PCT/US2019/051172 WO2020056370A1 (en) | 2018-09-13 | 2019-09-13 | Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| EP3863645A1 EP3863645A1 (en) | 2021-08-18 |
| EP3863645A4 true EP3863645A4 (en) | 2022-11-16 |
Family
ID=69778403
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP19861226.9A Pending EP3863645A4 (en) | 2018-09-13 | 2019-09-13 | Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20220054653A1 (en) |
| EP (1) | EP3863645A4 (en) |
| JP (1) | JP2022500443A (en) |
| AU (1) | AU2019338557A1 (en) |
| CA (1) | CA3111836A1 (en) |
| WO (1) | WO2020056370A1 (en) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11364292B2 (en) | 2015-07-21 | 2022-06-21 | Modernatx, Inc. | CHIKV RNA vaccines |
| MA42502A (en) | 2015-07-21 | 2018-05-30 | Modernatx Inc | VACCINES AGAINST INFECTIOUS DISEASE |
| WO2017031232A1 (en) | 2015-08-17 | 2017-02-23 | Modernatx, Inc. | Methods for preparing particles and related compositions |
| MD3718565T2 (en) | 2015-10-22 | 2022-09-30 | Modernatx Inc | Respiratory virus vaccines |
| EP3364980A4 (en) | 2015-10-22 | 2019-07-10 | ModernaTX, Inc. | Nucleic acid vaccines for varicella zoster virus (vzv) |
| WO2017070624A1 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Tropical disease vaccines |
| JP6921833B2 (en) | 2015-10-22 | 2021-08-18 | モデルナティーエックス， インコーポレイテッド | Human cytomegalovirus vaccine |
| CA3007955A1 (en) | 2015-12-10 | 2017-06-15 | Modernatx, Inc. | Lipid nanoparticles for delivery of therapeutic agents |
| MX2019002904A (en) | 2016-09-14 | 2019-09-26 | Modernatx Inc | High purity rna compositions and methods for preparation thereof. |
| EP3528821A4 (en) | 2016-10-21 | 2020-07-01 | ModernaTX, Inc. | Human cytomegalovirus vaccine |
| WO2018089851A2 (en) | 2016-11-11 | 2018-05-17 | Modernatx, Inc. | Influenza vaccine |
| EP3551193A4 (en) | 2016-12-08 | 2020-08-19 | Modernatx, Inc. | Respiratory virus nucleic acid vaccines |
| US11384352B2 (en) | 2016-12-13 | 2022-07-12 | Modernatx, Inc. | RNA affinity purification |
| WO2018170256A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Herpes simplex virus vaccine |
| MA52262A (en) | 2017-03-15 | 2020-02-19 | Modernatx Inc | BROAD SPECTRUM VACCINE AGAINST THE INFLUENZA VIRUS |
| WO2018170260A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Respiratory syncytial virus vaccine |
| WO2018170270A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Varicella zoster virus (vzv) vaccine |
| EP3595676A4 (en) | 2017-03-17 | 2021-05-05 | Modernatx, Inc. | Zoonotic disease rna vaccines |
| WO2018187590A1 (en) | 2017-04-05 | 2018-10-11 | Modernatx, Inc. | Reduction or elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins |
| WO2018232357A1 (en) | 2017-06-15 | 2018-12-20 | Modernatx, Inc. | Rna formulations |
| CN111212905A (en) | 2017-08-18 | 2020-05-29 | 摩登纳特斯有限公司 | RNA polymerase variants |
| MA49922A (en) | 2017-08-18 | 2021-06-02 | Modernatx Inc | PROCESSES FOR HPLC ANALYSIS |
| EP3668977A4 (en) | 2017-08-18 | 2021-04-21 | Modernatx, Inc. | Analytical hplc methods |
| CA3073211A1 (en) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | Methods of making lipid nanoparticles |
| WO2019055807A1 (en) | 2017-09-14 | 2019-03-21 | Modernatx, Inc. | Zika virus rna vaccines |
| US11911453B2 (en) | 2018-01-29 | 2024-02-27 | Modernatx, Inc. | RSV RNA vaccines |
| US11351242B1 (en) | 2019-02-12 | 2022-06-07 | Modernatx, Inc. | HMPV/hPIV3 mRNA vaccine composition |
| US11851694B1 (en) | 2019-02-20 | 2023-12-26 | Modernatx, Inc. | High fidelity in vitro transcription |
| AU2020224103A1 (en) | 2019-02-20 | 2021-09-16 | Modernatx, Inc. | Rna polymerase variants for co-transcriptional capping |
| CN111041025B (en) | 2019-12-17 | 2021-06-18 | 深圳市瑞吉生物科技有限公司 | mRNA targeting molecule based on combination of N-acetylgalactosamine polypeptide and preparation method thereof |
| CN115811986A (en) | 2020-04-22 | 2023-03-17 | 生物技术欧洲股份公司 | Coronavirus vaccine |
| CN111744019B (en) * | 2020-07-01 | 2023-08-04 | 深圳瑞吉生物科技有限公司 | Mannose-based mRNA targeted delivery system and application thereof |
| EP3936946A1 (en) | 2020-07-10 | 2022-01-12 | Patek Philippe SA Genève | Timepiece oscillator with flexible pivot |
| US11406703B2 (en) | 2020-08-25 | 2022-08-09 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| WO2024002985A1 (en) | 2022-06-26 | 2024-01-04 | BioNTech SE | Coronavirus vaccine |
| CN115404273B (en) * | 2022-06-30 | 2023-06-23 | 湖南家辉生物技术有限公司 | ATP8B1 gene mutant, protein and application of type I causing progressive familial intrahepatic cholestasis |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20140275229A1 (en) * | 2012-04-02 | 2014-09-18 | Moderna Therapeutics, Inc. | Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1 |
| WO2017001570A2 (en) * | 2015-06-30 | 2017-01-05 | Ethris Gmbh | Atp-binding cassette family coding polyribonucleotides and formulations thereof |
| WO2017075038A1 (en) * | 2015-10-26 | 2017-05-04 | Rana Therapeutics, Inc. | Nanoparticle formulations for delivery of nucleic acid complexes |
| WO2017201333A1 (en) * | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9012219B2 (en) * | 2005-08-23 | 2015-04-21 | The Trustees Of The University Of Pennsylvania | RNA preparations comprising purified modified RNA for reprogramming cells |
| RS63964B1 (en) * | 2005-08-23 | 2023-03-31 | Univ Pennsylvania | Rna containing modified nucleosides and methods of use thereof |
| WO2011025862A2 (en) * | 2009-08-28 | 2011-03-03 | Curna, Inc. | Treatment of atp-binding cassette sub-family b member 1 (abcb1) related diseases by inhibition of natural antisense transcript to abcb1 |
| WO2014062736A1 (en) * | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| US20160032316A1 (en) * | 2013-03-14 | 2016-02-04 | The Trustees Of The University Of Pennsylvania | Purification and Purity Assessment of RNA Molecules Synthesized with Modified Nucleosides |
| WO2017153936A1 (en) * | 2016-03-10 | 2017-09-14 | Novartis Ag | Chemically modified messenger rna's |
-
2019
- 2019-09-13 JP JP2021514132A patent/JP2022500443A/en active Pending
- 2019-09-13 WO PCT/US2019/051172 patent/WO2020056370A1/en unknown
- 2019-09-13 AU AU2019338557A patent/AU2019338557A1/en active Pending
- 2019-09-13 US US17/276,112 patent/US20220054653A1/en active Pending
- 2019-09-13 CA CA3111836A patent/CA3111836A1/en active Pending
- 2019-09-13 EP EP19861226.9A patent/EP3863645A4/en active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20140275229A1 (en) * | 2012-04-02 | 2014-09-18 | Moderna Therapeutics, Inc. | Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1 |
| WO2017001570A2 (en) * | 2015-06-30 | 2017-01-05 | Ethris Gmbh | Atp-binding cassette family coding polyribonucleotides and formulations thereof |
| WO2017075038A1 (en) * | 2015-10-26 | 2017-05-04 | Rana Therapeutics, Inc. | Nanoparticle formulations for delivery of nucleic acid complexes |
| WO2017201333A1 (en) * | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
Non-Patent Citations (1)
| Title |
|---|
| See also references of WO2020056370A1 * |
Also Published As
| Publication number | Publication date |
|---|---|
| EP3863645A1 (en) | 2021-08-18 |
| US20220054653A1 (en) | 2022-02-24 |
| CA3111836A1 (en) | 2020-03-19 |
| AU2019338557A1 (en) | 2021-03-25 |
| WO2020056370A1 (en) | 2020-03-19 |
| WO2020056370A9 (en) | 2020-05-07 |
| JP2022500443A (en) | 2022-01-04 |
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| RIC1 | Information provided on ipc code assigned before grant |
Ipc: A61P 1/16 20060101ALI20220707BHEP Ipc: C12N 15/67 20060101ALI20220707BHEP Ipc: A61K 31/7088 20060101ALI20220707BHEP Ipc: C12N 15/113 20100101ALI20220707BHEP Ipc: A61K 9/51 20060101ALI20220707BHEP Ipc: A61K 31/7115 20060101AFI20220707BHEP |
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| RIC1 | Information provided on ipc code assigned before grant |
Ipc: A61P 1/16 20060101ALI20221010BHEP Ipc: C12N 15/67 20060101ALI20221010BHEP Ipc: A61K 31/7088 20060101ALI20221010BHEP Ipc: C12N 15/113 20100101ALI20221010BHEP Ipc: A61K 9/51 20060101ALI20221010BHEP Ipc: A61K 31/7115 20060101AFI20221010BHEP |